KR20220004675A - 단축 센스 가닥을 갖는 이중 가닥 핵산 억제제 분자 - Google Patents

단축 센스 가닥을 갖는 이중 가닥 핵산 억제제 분자 Download PDF

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KR20220004675A
KR20220004675A KR1020217037233A KR20217037233A KR20220004675A KR 20220004675 A KR20220004675 A KR 20220004675A KR 1020217037233 A KR1020217037233 A KR 1020217037233A KR 20217037233 A KR20217037233 A KR 20217037233A KR 20220004675 A KR20220004675 A KR 20220004675A
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nucleotides
nucleic acid
length
acid inhibitor
sense strand
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KR1020217037233A
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English (en)
Korean (ko)
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밥 데일 브라운
웨이민 왕
네임 나제프
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다이서나 파마수이티컬, 인크.
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Publication of KR20220004675A publication Critical patent/KR20220004675A/ko

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217037233A 2019-05-03 2020-04-29 단축 센스 가닥을 갖는 이중 가닥 핵산 억제제 분자 KR20220004675A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962842908P 2019-05-03 2019-05-03
US62/842,908 2019-05-03
PCT/US2020/030398 WO2020226960A1 (fr) 2019-05-03 2020-04-29 Molécules d'inhibiteur d'acide nucléique double brin à brins sens raccourcis

Publications (1)

Publication Number Publication Date
KR20220004675A true KR20220004675A (ko) 2022-01-11

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Country Status (13)

Country Link
US (1) US20220177880A1 (fr)
EP (1) EP3963072A1 (fr)
JP (1) JP2022530678A (fr)
KR (1) KR20220004675A (fr)
CN (1) CN113795581A (fr)
AU (1) AU2020268798A1 (fr)
BR (1) BR112021021686A2 (fr)
CA (1) CA3136676A1 (fr)
CL (1) CL2021002842A1 (fr)
IL (1) IL287354A (fr)
MX (1) MX2021013418A (fr)
SG (1) SG11202111077RA (fr)
WO (1) WO2020226960A1 (fr)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022512578A (ja) 2018-10-09 2022-02-07 ザ ユニヴァーシティ オブ ブリティッシュ コロンビア 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法
WO2023081500A2 (fr) * 2021-11-08 2023-05-11 Dicerna Pharmaceuticals, Inc. Conjugués oligonucleotides/arni
WO2023207615A1 (fr) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 Classe de composés d'arni double brin comportant un débordement constitué de nucléotides naturels

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MX2021013418A (es) 2021-12-10
CA3136676A1 (fr) 2020-11-12
BR112021021686A2 (pt) 2022-03-22
CN113795581A (zh) 2021-12-14
AU2020268798A1 (en) 2021-11-04
SG11202111077RA (en) 2021-11-29
WO2020226960A1 (fr) 2020-11-12
CL2021002842A1 (es) 2022-06-10

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